Our Impact on Cancer Research

We’re committed to showcasing the exceptional work your donation enables — from sponsoring talented physicians and researchers, to supporting pediatric inpatient units and purchasing state-of-the-art equipment. Below is an overview of some of the projects and progress our donors make possible:

The Pediatric Tumor Biology Lab has made groundbreaking progress understanding the biology of pediatric solid tumors and unlocking anti-angiogenic therapies. PCF has been a lab supporter since 1999 and has donated millions of dollars, currently representing a third of the lab’s annual budget. With PCF’s support, the lab continues to perform significant preclinical work in the field of tumor angiogenesis and metastases. We have recently formed partnerships with Radiation Oncology and Immunology to use our mouse models of pediatric cancer to study how radiation affects the tumor vessels and how the immune system contributes to fighting cancer.

With PCF’s support, the lab performed significant preclinical work that eventually led to FDA approval for two of the first anti-angiogenic drugs. The lab enjoys a steady stream of young minds who spend time in the facility and share a commitment to curing cancer. Support from PCF has also helped the lab to educate more than 50 surgery residents, oncology residents, and medical students.

With PCF’s support, the Lab performed significant preclinical work that eventually led to FDA approval for two of the first anti-angiogenic drugs, bevacizumab (Avastin®) and aflibercept (Zaltrap®).

Together with peers at CUMC’s department of Obstetrics and Gynecology, Division of Maternal Fetal Medicine, the Lab has led the way in treating pregnant mothers whose fetuses are affected with a specific type of vascular malformation, called lymphatic malformations, with a heart medication that was unexpectedly found to be effective in shrinking this very difficult type of blood vessel tumor.

With support from PCF, the lab has educated more than 50 surgery residents, oncology residents, medical students, college students, and high school students in the student of angiogenesis (tumor blood vessel growth). Most of the Lab’s medical trainees are also conducting research in their own right, again propagating the fight against pediatric cancer.

The Lab has published more than 40 papers in the past 10 years in high impact peer reviewed journals.

University of Chicago Medicine Comer Children’s Hospital

PCF supports the work of Dr. Jessica Kandel, who specializes in treatment methods for recalcitrant tumors. Dr. Kandel’s early work at Columbia contributed to Genetech’s decision to sponsor clinical trials of the drug Avastin. Dr. Kandel was part of the clinical trial team for Avastin in children, and represented the first complete translation from bench-to-bedside. Avastin has gone on to become a widely used treatment for advanced cancers in both children and adults.

PCF is proud to have supported Dr. Kandel’s move to University of Chicago, allowing her team to tap into a whole different set of experts at a leading research institution. Her lab continues to focus on the role of abnormal blood vessel growth in childhood tumors. Dr. Kandel’s team recently found that a toxin made by a common bacteria produces changes in the blood vessels that affect metastasis. The group is particularly proud of the advances in patient care that this line of exciting research has made possible.

At Comer Children’s Hospital, the PCF-funded medical team has been researching how blood vessel growth impacts the spread and growth of tumors.“Tumors cannot grow unless they receive oxygen and nutrients from the bloodstream. In addition, the bloodstream provides a path for travel of cancerous cells to other sites in the body,” says Jessica Kandel, Surgeon-in-Chief at Comer Children’s Hospital. “For these reasons, the growing understanding of tumors’ control of blood vessels has yielded cutting-edge advances in cancer therapy. Our lab has focused on cancers of childhood, which have been understudied compared with adult cancers. We study tumors such as neuroblastoma, which remain deadly to most children diagnosed after infancy, and for whom current treatments have significant toxicities.”

According to Kandel, PCF supported research was critical in demonstrating that blocking certain blood vessel-stimulating molecules could suppress the growth of pediatric cancers. The research led to the successful clinical trials of groundbreaking drugs in children, and to increasing the array of options available to pediatric oncologists.

“Importantly, these projects involved not only basic investigation, but the training of future pediatric cancer investigators – of whom there are not enough,” says Kandel. “None of this work would have been possible without the steadfast support of PCF.”

Through the Pediatric Cancer Foundation Developmental Therapeutic Program, PCF funding has been instrumental in establishing and maintaining a robust clinical research program under the leadership of principal investigator Dr. Julia Glade-Bender. Dr. Bender’s work enables the translation of the newest findings from laboratory bench to patient bedside, and explores the utility and safety of these novel treatments through clinical trials for children with incurable cancer. The PCFDTP also serves as an incubator for precision cancer medicine — individually tailored therapies based on molecular and genetic characteristics.

Funding from PCF has allowed the program to build a strong core infrastructure with a research manager, research nurse practitioner, two clinical research associates, and a regulatory specialist. This base enables the program to accommodate more children with relapses or hard-to-treat cancers and open a greater diversity of trials:

The PCFDTP currently has 30 trials available for relapsed/refractory cancer. In addition, the program has implemented a next-generation Pediatric Oncology precision medicine program using genetic sequencing (PIPseq) to improve the care of individual children with cancer at Columbia.Columbia is the founding member of the elite National Cancer Institute (NCI) sponsored Children’s Oncology Group Phase I Consortium, one of only 21 institutions in North America, and the only site in the New York-New Jersey-Connecticut tri-state area. Because of Columbia’s record of achievement and care, pharmaceutical companies regard PCFDTP as a “center of excellence” for the conduct of industry-sponsored trials.

PCF has supported the careers of promising young physician scientists at this institution since 2010. Specifically, PCF support helps fund the three-year Hematology/Oncology Fellowship Program, where fellows are trained as scientist-clinicians dedicated to comprehensive, compassionate, and research-driven care. Fellows have accomplished amazing things, from clinical trials to seminal discoveries. PCF has also contributed funds to the installation of special privacy screens in infusion treatment rooms, which bolster patients’ quality of care.

PCF Fellows have forged a comprehensive effort to map the evolution of relapsed Acute Lymphoblastic Leukemia (ALL) by using sophisticated genomic technology to uncover differences in the genetic landscape of blasts at relapse that allow them to evade therapy. This work has led to two clinical trials and a potential third trial. This work has also enabled the detection of low level resistant cells before clinical relapse so that therapy can be altered to prevent relapse.

Investigators have used new drugs (“epigenetic” agents) that restore the normal epigenome (made up of the proteins that “insulate” DNA and control the expression of genes), thus eradicating brain tumors and making leukemia cells more sensitive to chemotherapy.

A PCF Fellow has determined that translocations (mutations in the DNA) do not occur randomly, but are based on the fact that chromosomes are distributed in a particular order within the nucleus of a cell. In developing normal lymphocytes, the location of chromosomes favor the occurrence of particular translocations that are frequently seen in childhood leukemia.

A PCF-funded Fellow discovered cells identified by the expression of a particular set of proteins that appears to be able to be responsible for making additional tumor cells. A drug already exists that can inactivate one of the proteins that are characteristic of osteosarcoma stem cells. Work is ongoing to determine whether the drug successfully leads to tumor cell death.

Memorial Sloan Kettering Cancer Center, Department of Pediatrics

Since 1997, PCF has provided more than $1.4 million to Memorial Sloan Kettering Cancer Center’s Department of Pediatrics. This generous funding has supported MSK’s dedicated physician-scientists, working tirelessly to develop immune-based therapy for pediatric solid tumor malignancies. Currently, funding from PCF is allowing MSK’s neuroblastoma team to conduct studies of next-generation antibody therapy—a therapeutic technology that holds incredible promise as a more effective treatment than chemotherapy or radiation with fewer long-term side effects. PCF’s consistent support for clinical trials is critical to reaching MSK’s shared goal of making safe, life-saving treatments available to all children facing a cancer diagnosis.

PCF has been partnered with Memorial Sloan Kettering Cancer Center (MSK) since 1997, providing funds that have allowed investigators at the largest pediatric oncology program in the United States to undertake new research initiatives.

Because of the rarity of pediatric cancers, pharmaceutical companies do not devote the resources to develop drugs for these diseases, while the National Cancer Institute only allocates 4% of its annual budget towards pediatric cancer.

Funding from PCF has allowed MSK’s Department of Pediatrics to build an infrastructure through which the latest technologies can be applied to develop new treatments to help children affected by cancer. Funding from PCF was used to support production costs for a new antibody therapy, developed at MSK by Nai-Kong Cheung, MD, PhD, to treat neuroblastoma – a devastating pediatric cancer. The close collaboration between our researchers and physicians makes it possible for us to quickly bring these lifesaving treatments from the lab bench to the patient bedside.

Northwell Health – Feinstein Institute for Medical Research

Dr. Jeff Lipton

PCF funds research by Drs. Jeff Lipton and Lio Blanc, who study which gene mutations give rise to malignancy in children. Drs. Jeffrey Lipton and Lionel Blanc note: “Without funding from PCF, we would not be able to expand our work, which aims to understand the pathogenesis of the most common pediatric bone cancer, osteogenic sarcoma. These funds have not only aided our work at the Feinstein Institute of Medical Research, but also fostered new long-term collaborations with outside institutions.”

PCF has been providing support to The Cohen Children’s Medical Center of New York for 15 consecutive years. The team has garnered major grants from the NIH, DOD and the CDC, all based upon work initially supported by PCF.

Research funded by PCF at The Feinstein Institute has been directed towards studying and understanding Diamond Blackfan anemia.

“Through decades of support from PCF, my group has been able to understand the biology of Diamond Blackfan anemia, connect the disease to cancer predisposition and develop animal and cellular models that we are now able to use to understand the biology of cancer,” says Jeffery Lipton, head of Patient Orient Research at The Feinstein Institute. Diamond Blackfan anemia is a rare childhood bone marrow failure syndrome that results in the inability to produce red blood cells. Lipton says the Feinstein research has linked the disease to an increased rate of myelodysplastic syndrome (MDS; pre-leukemia), osteogenic sarcoma, and gastrointestinal luminal cancers such as esophagus, stomach, colon, and rectum cancers.

According to Lipton, the biggest challenge in the way of pediatric cancer research is a large number of great scientific ideas put forward with not enough minds and funding to put the research into execution. Despite the difficulties the field faces, Lipton looks forward to an exciting year filled with new developments.

“Advances in targeted therapy based upon a remarkable growth in our understanding of cancer genetics and genomics will result in the development of new and effective targeted therapies, both with small molecules targeting the mutations that result in cancer, as well as immunologic approaches that enhance and modify the tumor microenvironment and the immune system itself to directly attack cancer,” says Lipton.

“Deaths from childhood cancer continue to decrease as researchers at all of the institutions supported by PCF make major strides in our efforts to prevent or cure all of childhood cancer. I look forward to continued support from PCF until we can all declare the battle won!”

Maria Fareri Children’s Hospital Westchester Medical Center

PCF funding has helped in the development of innovative treatments to battle hematologic malignancies in children and adolescents through targeted immunotherapies, novel chemotherapy for induction therapy, stem cell transplantation, and other methods. PCF funding has also made possible a pediatric infusion center, as well as a Nurse Practitioner Coordinator to support, coordinate, and assist in clinical research.

Pediatric Cancer Foundation’s mission is to develop a cure
for childhood cancer. For more than 45 years, the 501(c) (3)
nonprofit has raised money for game-changing research,
patient treatment and endowing world renowned doctors
at the hospitals PCF supports.